NIH’s first rare disease collaboration with Big Pharma

In Clinical Development, Partnering & Investment by Tim Peplow

national institutes of health (National Cancer Institute)

The National Institutes of Health (NIH) is taking on four new pre-clinical drug development projects, including its first collaboration with Big Pharma.

The NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program will support an Eli Lilly project to develop a potential treatment for hypoparathyroidism. The three remaining projects include two approaches to treating retinitis pigmentosa, and the treatment of hypertrophic cardiomyopathy  in patients with LEOPARD syndrome.

The Eli Lilly project will examine the use of a long-acting parathyroid hormone analog for the treatment of hypoparathyroidism, which can lead to cardiac problems and convulsions.

Through the TRND, the NIH’s National Center for Advancing Translational Sciences (NCATS) is looking to “de-risk” the development of treatments for rare diseases, an avenue which private companies often do not explore due to a perceived low return on investment. “TRND is grounded in partnerships with academic, government, pharmaceutical and patient advocacy groups. Working in collaboration, scientists conduct pre-clinical development of new drugs and then advance them to first-in-human clinical trials,” said NCATS Director Christopher P. Austin, M.D. In the last two years, the program has advanced treatments for sickle cell disease, chronic lymphocytic leukaemia, hereditary inclusion body myopathy, and Niemann-Pick Type C.

Read the press release >

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